Project Grant R01HL187977
- The National Institutes of Health National Heart Lung and Blood Institute awarded Sanford Burnham Prebys Medical Discovery Institute $1,603,648 on July 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop a single-construct gene therapy for enhanced cardiac repair. The funded research aims to overcome barriers to cardiac reprogramming by creating a gene therapy that simultaneously induces overexpression of cardiac transcription factors MEF2C, GATA4, and TBX5 (MGT)...
- The National Heart, Lung, and Blood Institute awarded Duke University $814,943 on July 15, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop non-viral gene therapy approaches for cardiac regeneration following myocardial injury. Duke University will develop cardiac-targeted lipid nanoparticles for in vivo transfection of cardiomyocytes and adapt a cell-state sensing platform (CELLREADR) to deliver therapeutic transgenes with cell-type precision to injured...
- The National Heart, Lung, and Blood Institute awarded Case Western Reserve University, Office of Research Administration $1,532,398 on July 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop and validate a gene therapy delivery platform for cardiomyopathies. The project, titled "Bait and Switch Gene Therapy for Cardiomyopathies," addresses the clinical translation barrier in adeno-associated virus (AAV) gene therapy for genetic heart diseases including...
- The National Heart, Lung, and Blood Institute awarded the University of California, San Diego $779,600 on July 10, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate the developmental mechanisms that direct cardiac cell diversification and left ventricle morphogenesis. The research employs single-cell transcriptomic analyses and lineage tracing in mouse embryos to define the gene regulatory networks controlling how diverse cardiac cell types emerge from...
- The National Heart, Lung, and Blood Institute awarded the University of California, Davis $590,503 on August 5, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to conduct research on cardiac regenerative therapy using gene-edited stem cells to improve transplantation outcomes in end-stage heart failure. The research addresses inflammation-mediated cell death that impedes cardiac stem cell therapy by modifying both donor cells and the host environment to target genes...
- The National Heart, Lung, and Blood Institute awarded $599,669 to Kinea Bio Inc. on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop and validate KNA-123, an innovative gene therapy for Duchenne muscular dystrophy targeting cardiac muscle degeneration. The award is classified as a Project Grant. Kinea Bio will leverage the split intein-mediated protein ligation for gene therapy (SIMPLI-GT) platform to enable delivery of a larger, more functional...
- The National Heart, Lung, and Blood Institute awarded $1,137,435 to the University of Pennsylvania on March 20, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to advance therapeutic gene-editing approaches for cardiovascular and inherited disease. The recipient is developing and translating gene-editing therapies across three therapeutic domains. The first targets liver-centered, multiplex editing to prevent atherosclerotic cardiovascular disease through tunable...
- The National Heart, Lung, and Blood Institute awarded The Leland Stanford Junior University $2,339,136 on May 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate the role of phospholamban in regulating perinuclear calcium signaling and pathological cardiac gene expression as a potential therapeutic target for dilated cardiomyopathy. The funded research examines how phospholamban interacts with the scaffold protein AKAP6B at the myocyte outer nuclear...
- The National Heart, Lung, and Blood Institute awarded University of California Irvine $711,796 on July 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop a nanomaterial-engineered proteomic platform for mapping curvature-dependent cardiac signaling. The project delivers a nanopro (nanomaterial-engineered proteomic) platform that uses nanostructures to perform proximity-labeling of nearby proteins on cardiomyocyte cell membranes, producing unbiased,...
- The National Heart Lung and Blood Institute awarded The Trustees of the University of Pennsylvania $623,909 on August 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to support rational design of gene therapy transgenes for Duchenne muscular dystrophy. The project addresses immunogenicity and incomplete functionality of miniaturized dystrophin transgenes in adeno-associated virus vector–based approaches to DMD gene therapy. The research employs a structure-guided...
The National Heart, Lung, and Blood Institute awarded the University of California, San Diego $795,900 on August 25, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop gene therapy for FLNC haploinsufficiency cardiomyopathy. FLNC haploinsufficiency, caused by nonsense, frameshift, or splice-site mutations, drives inherited dilated and arrhythmogenic cardiomyopathies characterized by early-onset systolic dysfunction, high malignant arrhythmia risk, and sudden cardiac death. Current therapies relieve symptoms or reduce arrhythmia risk but do not restore FLNC protein levels and thus fail to halt disease progression. Gene replacement therapy offers a mutation-agnostic strategy to address the underlying cause, but full-length FLNC complementary DNA (~8.2 kilobases) far exceeds the packaging capacity of adeno-associated virus vectors (~4.7 kilobases), making traditional single-vector delivery unfeasible. Existing technologies like split-intein and stitchr can reconstitute large transgenes from multiple vectors but each has technical limitations. The funded research engineered a truncated version termed miniflnc (~5.7 kilobases) by deleting the IG11-IG19 subdomain. Homozygous miniflnc mice display normal cardiac structure and function, indicating this shorter version is sufficient for cardiac development and adult cardiac homeostasis maintenance. Performance occurs in La Jolla, California. The award period extends from August 25, 2026, through May 31, 2030.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $795.9k | 8/25/26 |